Four ways patient advocates help drive research

Last week I attended the Stem Cell Meeting on the Mesa, an annual event organized by CONNECT. The meeting included all the stellar scientific panels I expected and one I didn’t expect: “Patient Advocacy 2.0 – Can they participate?”

The panel discussed opportunities for patient participation and the ethics involved. I was captivated by panel member Dani Grady’s story of surviving breast cancer and her advocacy for increased cancer research funding, education, improved patient care and more patient participation in clinical trials. It was interesting to hear how a patient’s perspective can improve clinical trials and the drug approval process. But as I sat there, I couldn’t help wondering… how can patients participate in basic research – the earliest phase of biomedical discovery, during which the molecular underpinnings of disease are only just beginning to be understood?

So I did a little research of my own.

It turns out that stem cells are one area in particular where patient advocates can help drive research and funding. In fact, the California Institute for Regenerative Medicine (CIRM), the $3 billion stem cell agency created after California voters approved ballot measure Prop 71 in 2004, is mandated by the state to involve patient advocates. CIRM makes grants and provides loans for stem cell research and is led by a governing body that includes 12 patient advocates representing nine disease areas.

In a recent Nature Medicine article, CIRM governing board member James Sheehy writes, “The presence of vocal, engaged patient advocates has added an indispensable dimension to the proceedings. In measuring research quality, advocates tend to focus on a project’s ability to benefit people—not just drive scientific curiosity—which keeps even basic biomedical research grounded in its ability to produce concrete health benefits.”

Here are four specific ways I think patient advocates can help drive basic research:

1. Informing the public

Stem cell research is a good example of one area where patient advocates help drive basic discoveries. It’s probably safe to say that controversy, debate, politics and court rulings have hampered this work. That’s why patient advocates need to work side-by-side with scientists to inform the public, win public support and lobby for stem cell funding.

Need proof? In the same Nature Medicine article, Sheehy points out that “CIRM and its billions of dollars would not exist without the work of patients in convincing the people of California to support the venture.”

2. Driving new research directions

Sometimes patients – the people actually experiencing the symptoms – are the only ones who can put together all the pieces of a puzzling disease. Several years ago, Dr. Yu Yamaguchi was studying bone development when he received a call from Sarah Ziegler. Ziegler’s son, Robert, suffers from a rare inherited bone disorder called multiple hereditary exostoses (MHE). Over time, as Robert endured many surgeries to treat an incurable disease, Ziegler dug deep into the scientific literature to educate herself about MHE and bones. Somewhere along the way, she came across Dr. Yamaguchi’s work and had a feeling his research could impact MHE.

One of Ziegler’s greatest frustrations was that most medical specialists only saw one part of Robert’s disease, when she knew from her experience with him and other MHE patients that the disease affects more than just bones.

In a letter recounting her eventual friendship with Dr. Yamaguchi, Ziegler recalls, “Beyond bone deformities, I had seen that MHE patients shared many other symptoms. [Dr. Yamaguchi and I] discussed the orthopedic aspects as well as non-orthopedic effects/symptoms that people with MHE experience and the reasons orthopedic surgeons had not picked up on many of these.”

This friendship built on advocacy recently bore fruit: Earlier this year, Dr. Yamaguchi’s team developed a mouse model of MHE, a much-needed tool that will allow researchers to better study the disease and develop new therapies.

3. Funding high-risk research

Most academic research is funded by grants from government agencies like the National Institutes of Health (NIH). However, most governmental funding agencies hesitate to fund “high-risk” research–great ideas that don’t have enough preliminary data to “warrant” funding. Of course, it’s just this type of high-risk research that has the potential to yield big results. In many cases, patient advocacy groups have stepped in to help fund very early research that otherwise might not get off the ground.

A few years ago, Dr. Pamela Itkin-Ansari received a JDRF Innovative Grant that gave her just one year’s worth of funding. (She calls it the “we-think-you-might-be-crazy-but-we’re-going-to-give-you-a-chance grant.”) This was the first funding she received to transplant diabetic mice with insulin-producing cells that are protected from immune destruction by an encapsulation device. The results from that JDRF-funded study were promising enough for her to apply successfully for CIRM funding. Now her work on cell transplantation without immunosuppression has moved through pre-clinical trials.

4. Providing personal motivation

Let’s be honest – working day and night in the laboratory can be a tough life. After hours and hours at the bench, writing grants and publishing papers, it’s easy for scientists to lose sight of the end goal: improving lives. While I was working on another blog post a few months ago, Dr. Lorenzo Puri told me how much it means to him to stay connected to patients suffering from the disease he studies – muscular dystrophy.

“There’s an emotional charge in my lab,” he says. “We know there are children waiting for us to do our jobs, and when we work we are targeting those faces, those smiles.”

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At last week’s Stem Cell Meeting on the Mesa, Duane Roth, CONNECT CEO and patient advocacy panel moderator, asked how many people in the audience have families affected by disease. Every single person raised his or her hand. In the end, doesn’t that make us all patient advocates? I look forward to watching as more patients and patient advocacy groups form bonds with researchers to help drive the search for new understanding and new therapies here at Sanford-Burnham and in the greater scientific community.

Watch the Stem Cell Meeting on the Mesa patient advocacy panel in its entirety below (video by The Science Network):

Tell us why you think patients should play a bigger role in research and healthcare policy. What’s your story? Please leave a comment below.

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Beaker is the science blog of Sanford Burnham Prebys Medical Discovery Institute (SBP). Here, we share Institute and research news, scientist and leadership profiles, industry trends, as well as interviews with interesting people. Join the conversation by commenting on our blog posts and by sharing Beaker stories on your own social media channels. #TheNewBeaker